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                          01

                          Fi Europe 2024
                          Not Confirmed
                          Fi Europe 2024
                          Not Confirmed

                          Details : Koselugo (selumetinib) blocks MEK1/2, which are involved in stimulating cells to grow. It is approved for the treatment of symptomatic, inoperable PNs in aged 2 years and above.

                          Brand Name : Koselugo

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          November 12, 2024

                          Lead Product(s) : Selumetinib

                          Therapeutic Area : Oncology

                          Highest Development Status : Approved

                          Sponsor : Merck & Co

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          02

                          Fi Europe 2024
                          Not Confirmed
                          Fi Europe 2024
                          Not Confirmed

                          Details : Ultomiris (ravulizumab) is approved in the US as the first long-acting C5 complement inhibitor for patients with anti-aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder.

                          Brand Name : Ultomiris

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          March 25, 2024

                          Lead Product(s) : Ravulizumab

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved

                          Sponsor : AstraZeneca

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          03

                          Fi Europe 2024
                          Not Confirmed
                          Fi Europe 2024
                          Not Confirmed

                          Details : The collaboration aims to develop an undisclosed initial therapeutic molecule that applies JCR’s proprietary J-Brain Cargo®, BBB penetration technology, for a neurodegenerative disease.

                          Brand Name : Undisclosed

                          Molecule Type : Undisclosed

                          Upfront Cash : Undisclosed

                          March 19, 2024

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Neurology

                          Highest Development Status : Discovery

                          Recipient : JCR Pharmaceuticals

                          Deal Size : Undisclosed

                          Deal Type : Collaboration

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                          04

                          Fi Europe 2024
                          Not Confirmed
                          Fi Europe 2024
                          Not Confirmed

                          Details : AG-10 (acoramidis) is an investigational, orally-administered small molecule stabilizer of transthyretin (TTR), evaluated in Phase 3 trials for Transthyretin Amyloid Cardiomyopathy treatment.

                          Brand Name : AG10

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          February 02, 2024

                          Lead Product(s) : Acoramidis Hydrochloride

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Recipient : BridgeBio Pharma

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          05

                          Fi Europe 2024
                          Not Confirmed
                          Fi Europe 2024
                          Not Confirmed

                          Details : Under the agreement, Alexion expand its rare disease gene therapy portfolio by gaining Pfizer’s early-stage preclinical gene therapy programmes. The agreement furthers Alexion's commitment to advancing next-generation genomic medicines.

                          Brand Name : Undisclosed

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          September 20, 2023

                          Lead Product(s) : Gene Therapy

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Preclinical

                          Recipient : Pfizer Inc

                          Deal Size : $1,000.0 million

                          Deal Type : Licensing Agreement

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                          06

                          Fi Europe 2024
                          Not Confirmed
                          Fi Europe 2024
                          Not Confirmed

                          Details : The collaboration aims to identify novel drug targets for rare neurodegenerative and neuromuscular diseases by leveraging CONVERGE, a full-stack platform that combines highly predictive human tissue datasets with machine learning to find new targets.

                          Brand Name : Undisclosed

                          Molecule Type : Undisclosed

                          Upfront Cash : $42.0 million

                          September 08, 2023

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Neurology

                          Highest Development Status : Discovery

                          Recipient : Verge Genomics

                          Deal Size : $882.0 million

                          Deal Type : Collaboration

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                          07

                          Fi Europe 2024
                          Not Confirmed
                          Fi Europe 2024
                          Not Confirmed

                          Details : Under the agreement, Alexion will expand its rare disease gene therapy portfolio by gaining Pfizer’s early-stage preclinical gene therapy programmes. The agreement furthers Alexion's commitment to advancing next-generation genomic medicines.

                          Brand Name : Undisclosed

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          July 28, 2023

                          Lead Product(s) : Gene Therapy

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Preclinical

                          Recipient : Pfizer Inc

                          Deal Size : $1,000.0 million

                          Deal Type : Licensing Agreement

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                          08

                          Fi Europe 2024
                          Not Confirmed
                          Fi Europe 2024
                          Not Confirmed

                          Details : AG10 (acoramidis) is an investigational, orally-administered small molecule designed to potently stabilize tetrameric transthyretin (TTR), being investigated for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM).

                          Brand Name : AG10

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          July 17, 2023

                          Lead Product(s) : Acoramidis Hydrochloride

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase III

                          Recipient : BridgeBio Pharma

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          09

                          Fi Europe 2024
                          Not Confirmed
                          Fi Europe 2024
                          Not Confirmed

                          Details : ALXN2040 (danicopan) is an investigational oral medicine in development as an add-on to standard of care C5 inhibitor therapy Ultomiris (ravulizumab) or Soliris (eculizumab) for patients with PNH who experience clinically significant EVH.

                          Brand Name : ALXN2040

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          June 09, 2023

                          Lead Product(s) : Danicopan,Eculizumab,Ravulizumab

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase III

                          Recipient : AstraZeneca

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          10

                          Fi Europe 2024
                          Not Confirmed
                          Fi Europe 2024
                          Not Confirmed

                          Details : Under the agreement, the two companies will begin research and development of new treatments using the J-Brain Cargo®, blood-brain barrier (“BBB”) penetration technology, in combination with an undisclosed effector molecule for the treatment of a ne...

                          Brand Name : Undisclosed

                          Molecule Type : Undisclosed

                          Upfront Cash : Undisclosed

                          April 03, 2023

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Neurology

                          Highest Development Status : Discovery

                          Recipient : JCR Pharmaceuticals

                          Deal Size : Undisclosed

                          Deal Type : Collaboration

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